Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs

Spinal muscular atrophy (SMA) is a rare, inherited neuromuscular disease caused by deletion and/or mutation of the Survival of Motor Neuron 1 (SMN1) gene. A second gene, SMN2, produces low levels of functional SMN protein that are insufficient to fully compensate for the lack of SMN1. Risdiplam (RG7...

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Vydáno v:Pharmacol Res Perspect
Hlavní autoři: Poirier, Agnès, Weetall, Marla, Heinig, Katja, Bucheli, Franz, Schoenlein, Kerstin, Alsenz, Jochem, Bassett, Simon, Ullah, Mohammed, Senn, Claudia, Ratni, Hasane, Naryshkin, Nikolai, Paushkin, Sergey, Mueller, Lutz
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2018
Témata:
Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6262736/
https://ncbi.nlm.nih.gov/pubmed/30519476
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/prp2.447
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